Prevention of transcriptional gamma-globin gene silencing by inducing the hereditary persistence of fetal hemoglobin point mutation using chimeraplast-mediated gene targeting

ABSTRACT

Objective: Hemoglobin F [HbF] augmentation is considered a clinically beneficial phenomenon in beta-hemoglobinopathies. Prevention of gamma-globin gene silencing, inspired by the hereditary persistence of fetal hemoglobin, may be a suitable strategy to upregulate HbF expression in these patients. Therefore, our objective was to assess the potential feasibility of induced -117 G?A substitution in HBG promoter in prevention of transcriptional silencing of the gamma-globin